rare diseases

About 350 million people suffer from rare diseases, most of which are genetic. But the individual disorders affect relatively few people, so there’s little commercial incentive to bring these treatments to market.

Alex Yiu was born a seemingly healthy baby. But by age 2, his muscle control and speech were deteriorating. His baffling condition took a decade to diagnose. The reanalysis of a DNA test was the key.

President Donald Trump wants to streamline the U.S. Food and Drug Administration so people with rare diseases can get medication faster. But one Florida...

Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives by doing so. But nearly a third of the medicines aren't new or were repurposed many times for financial gain.

You often hear about children with rare afflictions that can’t be helped. Here are some young people who beat the odds:A 2-year-old with Shone's syndrome,…